A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).

PHASE2TerminatedINTERVENTIONAL
Enrollment

21

Participants

Timeline

Start Date

August 19, 2015

Primary Completion Date

September 24, 2018

Study Completion Date

September 24, 2018

Conditions
Spinal Muscular Atrophy
Interventions
DRUG

Nusinersen

Administered by intrathecal injection.

PROCEDURE

Sham Procedure

Small needle prick on the lower back at the location where the IT injection is normally made.

Trial Locations (7)

21287

The Johns Hopkins Hospital, Baltimore

55101

Gillette Children's Specialty Healthcare, Saint Paul

75235

The University of Texas Southwestern Medical Center, Dallas

80337

LMU-Campus Innenstadt, München

98105

Seattle Children's Research Institute, Seattle

90095-8344

David Geffen School of Medicine at UCLA, Los Angeles

06106

Connecticut Childrens Medical, Hartford

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Biogen

INDUSTRY